St. Jude and Mustang reported late Wednesday that their gene therapy led to fully functioning immune systems in newly diagnosed infants with X-linked severe combined immunodeficiency (SCID-X1) who were treated in the Phase I/II LVXSCID-ND...

The European Commission began a formal investigation of Aspen Pharmacare Holdings Ltd. (JSE:APN) amid concerns that the company breached EU antitrust rules by excessively raising the prices of five off-patent cancer medicines: chlorambucil, melphalan, mercaptopurine,...

bluebird amended the open-label, U.S. Phase I HGB-206 trial evaluating IV LentiGlobin BB305 to increase production of therapeutic anti-sickling hemoglobin and increase enrollment to up to 29 patients from up to 8. The changes include...

RxMP Therapeutics LLC is developing microparticles harvested from red blood cells to mitigate excessive bleeding during surgery or trauma by promoting clotting at sites of bleeding without instigating unwanted coagulation. Available hemostatics used for acute...

Data presented at the European Hematology Association meeting this month suggest both bluebird bio Inc. and Acceleron Pharma Inc. could have the first two disease-modifying treatments for beta thalassemia. While bluebird's gene therapy approach could...

Pharmalink said FDA granted Orphan Drug designation for Busulipo, a liposome formulation of the generic chemotherapeutic busulfan, as a conditioning agent for use in cancer patients prior to hematopoietic stem cell transplantation (HSCT). The company...

Pharmalink AB (Stockholm, Sweden) raised SEK96 million ($15 million) in a series C round co-led by new investor Investinor and existing investor Industrifonden. The company's founders, who are existing investors, also participated. Next year, Pharmalink...